Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 274 |
| INN | Gefurulimab |
| Status | Clinical |
| Drug code(s) | ALXN1720 |
| Brand name | None |
| mAb sequence source | mAb humanized |
| General Molecular Category | Bispecific |
| Format, general category | Fragment |
| Format details | sdAb, VHH-VHH' |
| Isotype (Fc) | None |
| Light chain isotype | None |
| Linker | None |
| Ave. DAR | None |
| Conjugated/fused moiety | None |
| Discovery method/technology | Camelid-derived |
| Target(s) | Complement C5, Albumin |
| Indications of clinical studies | Generalized Myasthenia Gravis, Proteinuria, Phase 1 in healthy volunteers |
| Primary therapeutic area | Immune-mediated / inflammatory disorders |
| Most advanced stage of development (global) | Phase 3 |
| Status | Active |
| Start of clinical phase (IND filing or first Phase 1) | September 04, 2019 |
| Start of Phase 2 | |
| Start of Phase 3 | October 15, 2022 |
| Date BLA/NDA submitted to FDA | |
| Year of first approval (global) | None |
| Date of first US approval | |
| INN, US product name | None |
| US or EU approved indications | None |
| Company | AstraZeneca |
| Licensee/Partner | None |
| Comments about company or candidate | NCT06607627 Phase 3 study in Generalized Myasthenia Gravis due to start in Mar 2025. Gefurulimab was granted orphan drug designation by the FDA for the treatment of patients with gMG. (September 2023) Phase 3 study for Generalized myasthenia gravis (NCT05556096) started in Nov 2022 is recruiting as of June 7, 2023 According to company website, 7 of nine cohorts are complete in a Phase 1 healthy volunteer study of ALXN1720. Due to COVID-19, the study was temporarily paused but is planned to restart in the third quarter of 2020. AstraZeneca acquired Alexion (formerly Syntimmune) NCT04920370 Phase 1 started in Sep 2019. |
| Full address of company | Cambridge, United Kingdom Europe United Kingdom https://www.astrazeneca.com/our-company/contact-us.html |
ALXN1720 is a novel anti-C5 albumin-binding bi-specific mini-body optimized for sub-cutaneous delivery that binds and prevents activation of human C5. MW = 25 kDa; VH - VH' Heavy-chain variable region antigen-binding fragment (VHH) antibodies targeting C5 and human serum albumin (HSA) were isolated from llama immune-based libraries and humanized. Gefurulimab comprises an N-terminal albumin-binding VHH connected to a C-terminal C5-binding VHH via a flexible linker. https://doi.org/10.1016/j.molimm.2023.12.004
| Anticipated events | None |
| Factor(s) contributing to discontinuation | None |